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Gene Therapy and Pharmacokinetics

Camargo

How and When to Incorporate PK Design into Your Gene Therapy Development Plan. Gene therapy, which was in its infancy around 30 years ago, is now becoming a more prominent treatment method in many therapeutic areas, from personalized therapy to mass vaccinations against COVID-19. Gene Therapy Definition.

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US FDA grants Fast Track status for Avidity’s AOC 1020 to treat FSHD

Pharmaceutical Technology

AOC 1020 has been designed for the treatment of the underlying cause of FSHD, which is caused by the abnormal expression of a gene known as double homeobox 4 or DUX4. Avidity Biosciences’ AOC 1020 is intended to reduce the DUX4 mRNA and DUX4 protein expression in muscles in these patients.

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Study highlights role of disordered protein interactions in gene expression

Scienmag

A team led by researchers at Baylor College of Medicine and the Czech Academy of Sciences has uncovered a new piece of the puzzle of how gene expression is orchestrated. Published in the journal Science, the findings reveal a novel mechanism that coordinates the assembly of components inside cells that control gene expression.

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Gene editing: beyond the hype

pharmaphorum

Cutting edge’ is, for once, a truly apt description when it comes to gene editing – both because the field is pushing medicine into areas we might never have dreamed possible, and because these technologies involve literally cutting DNA at a specific point in the genome. Zinc fingers. The exact mechanism depends on the disease in question.

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Scientists show how gene expression controls synaptic plasticity in the aging human brain

Medical Xpress

Scientific evidence shows how the cognitive decline in Alzheimer's disease (AD) is caused by the buildup of amyloid beta proteins, which promote synaptic malfunction.

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Alnylam identifies gene to lower abdominal fat and decrease cardiovascular risk

Pharmaceutical Technology

Alnylam Pharmaceuticals and collaborators have identified rare mutations in the INHBE gene that is expressed in the liver, related to a lower waist-to-hip ratio for body mass index (BMI), which is often used as an indicator of abdominal fat and is correlated with the risk of type 2 diabetes (T2D) and coronary heart disease.

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Salt stress alters legume responses to symbiotic rhizobacteria by modulating gene expression

Scienmag

Crop legumes are an integral part of sustainable agriculture, as several of these species represent an important protein source for both human and animal populations. Legumes engage in a unique and beneficial interaction with a group of soil bacteria, collectively called rhizobia.